Evaluation of Fluid-Based and Imaging Biomarkers for Interventional Trials in PSP

Principal Investigator: Dr. Mikael Simons, German Center for Neurodegenerative Diseases (DZNE), Munich, Germany

Treatment options for PSP remain limited to symptomatic treatment only with no cure available. Disease modification trials for PSP are limited by a lack of in vivo biomarkers both to confirm the diagnosis ante mortem and as an objective measure of whether the treatment is working. This project will investigate two such measures. One is neurofilament light chain (NfL), a protein in blood and CSF that has shown great promise in tracking the rate of neuronal death in PSP. The other is glial fibrillary acidic protein (GFAP), an newer biomarker for other neurodegenerative diseases that has yet to be investigated in PSP. GFAP measures over-abundance of astrocytes, a phenomenon related to disease severity. We will analyze NfL and GFAP in baseline and longitudinal samples of plasma and CSF samples of an exceptionally large PSP cohort. This cohort has detailed clinical and MRI imaging data available, allowing for comprehensive analysis of these biomarkers regarding their diagnostic and prognostic value in PSP. We will also investigate [18F] PI-2620 tau-PET showing the underlying 4RT-pathology and correlate the findings with the NfL and GFAP results.